Last update 20 Mar 2025

PLX-200

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Target
TPP1
Action
stimulants, inhibitors
Mechanism
TPP1 stimulants(Tripeptidyl Peptidase 1 stimulants), Apoptosis inhibitors
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Neuronal Ceroid-LipofuscinosesTay-Sachs DiseaseSandhoff Disease
Inactive Indication
Leukodystrophy, Globoid Cell
Originator Organization
Rush University Medical CenterRush University Medical Center
Active Organization
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
Inactive Organization-
Drug Highest PhasePhase 3
First Approval Date-
RegulationFast Track (United States), Orphan Drug (European Union)
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Related

1
Clinical Trials associated with PLX-200
NCT04637282
/ Not yet recruitingPhase 3
A Randomized. Multicenter, Double-Blind, Placebo-Controlled Safety, Tolerability, and Efficacy Study of PLX-200 in Participants With Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease
100 Clinical Results associated with PLX-200
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100 Translational Medicine associated with PLX-200
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100 Patents (Medical) associated with PLX-200
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100 Deals associated with PLX-200
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Neuronal Ceroid-LipofuscinosesPhase 3-
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
01 Dec 2024
Leukodystrophy, Globoid CellPhase 1
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
17 Aug 2021
Tay-Sachs DiseasePreclinical
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
28 Sep 2021
Sandhoff DiseasePreclinical
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
27 Aug 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

No Data

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Approval

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Regulation

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